July 22, 2021 : Aligning on Rare Drug Strategy, PMPRB, and Federal Election Engage with our Expert Panel in a Townhall style meeting to add your voice on these important issues in preparation for upcoming federal election. Panelist Alexandra Chambers (Novartis) Peter Brenders (BeiGene) Angela Genge (The Neuro) Fred Horne (3Sixty Public Affairs) Leanne Ward (CHEO)

1. “SORRY TO INTERRUPT
SUMMER HOLIDAYS”
Webinar Series
Webinar 1: Aligning on Rare Drug Strategy,
PMPRB, and Federal Election
July 22, 2021

2. “SORRY TO INTERRUPT
SUMMER HOLIDAYS”
Webinar Series
Webinar 2: Engaging Political Parties, Politicians,
and Policy Makers on Patient Needs
July 29, 2021

3. • Reading the Tea Leaves on federal government’s vision for Canada’s
“Expensive Drugs for Rare Diseases Strategy”: What can we surmise from
Health Canada’s read-out on “What We Heard” during the consultations.
• Playing “Whack a Mole” with PMPRB: DOWN with implementation of new
guidelines (3rd six-month hiatus); UP pops “out of the blue” pricing
restrictions on grandfathered drugs.
• SURPRISE! (Not). Federal election this fall. How do we align to align all
political parties and political candidates to support “rare disease” and “rare
drug” strategy (and safeguard the $500 million annual commitment)
Welcome and Brief Overview
Durhane Wong-Rieger, CORD
Bill Dempster, 3Sixty Public Affairs
Three BIG Issues

4. • Canada is one of the only developed countries
that does not have a national plan for rare
disease medicines
• 2019 federal budget includes a commitment to
invest in a national strategy for rare disease
treatments: up to $1 billion over two years
starting in 2022-23, with up to $500 million per
year afterwards
• 2020 fall economic statement and Speech from
the Throne reaffirmed this commitment
• National support for better access to rare
disease medicines has been building steadily
since 2015 and CORD’s launch of Canada’s Rare
Disease Strategy
• Both major national parties – Liberals /
Conservatives included rare disease drug
strategy in 2019 election platforms
Context and Opportunity

5. 1. Improving early detection and prevention,
2. Providing timely, equitable and evidence-informed care,
3. Enhancing community support,
4. Providing sustainable access to promising therapies and
5. Promoting innovative research
Rare Disease Strategy
- Five-point Action Plan

6. CORD’s 12-Steps to a National
Rare Disease Framework
• Patient empowerment: Empower patient organizations and patient
advocates as active full partners
• Creation of a Canadian Rare Drug Agency: independent, transparent,
publicly accountable agency with responsibility for all aspects of the
review of drugs for rare diseases, in coordination with Health Canada
• Create R&D incentives: Invest in Research and Development to support
therapeutic product accessibility, monitoring, and evaluation. Build
capacity for drug discovery, technological innovation, manufacturing and
production, and (global) distribution.
• Speed up access to treatment: Ensure timely availability of new
treatments by establishing a competitive and viable environment,
including supportive mechanisms for clinical trials, early access
programs, clinical site development, patient registries, and patient
support programs

7. CORD’s 12-Steps to a National
Rare Disease Framework
• Address regulatory barriers: Ensure PMPRB guidelines do not exceed
a “reasonable” threshold of fairness compared to comparable
countries; roll back 2019 PMPRB regulatory changes by removing use
of economic factors
• Improve regulatory approvals process: Ensure Health Canada
continues to update its regulatory process to encourage clinical trial
and new drug submissions for rare disease drugs
• Ensure pathways for special cases: For urgent need, timely access
provided through Special Access Program (SAP) prior to Health Canada
approval; create pathway through Early Access Programs.
• Need for multiple funding options: Multiple separate pathways
based on population size, disease severity, unmet need, evidence
uncertainty, potential therapeutic value, budget impact, annual unit
price, and industry

8. CORD’s 12-Steps to a National
Rare Disease Framework
• Leverage Managed Access Programs (MAPs): For drugs receiving
an NOC-C where there is uncertainty about the evidence at the time
of approval but where the unmet needs and benefits outweigh the
risks.
• Facilitate concurrent Health Canada and HTA reviews: Joint
Health Canada and HTA application (where appropriate)
• Support real-world evidence generation: Be responsible for real-
world monitoring, data collection, evaluating benefits, risks, and
uncertainty
• Enhance centres of clinical expertise: Partner in developing
Networked Centres of Expertise for specific rare diseases related to
management of a therapy.

9. Collaborative Canadian Network for
Rare Diseases (CCN4RD)
• Nation-wide, cross-disease, cross-disciplinary,
patient-inclusive network (physical and virtual) to
support optimal use of Rare Disease Treatments,
including locus of Real-World Data collection and
management
• National hub within WHO-RDI Collaborative
Global Network for Rare Diseases
Canada Rare Drug Strategy:
Pre-Election Activities
Workstreams
Health Technology Assessment for
Rare Disease Treatments
• Appropriate, consistent, transparent, and inclusive
framework and methodology for assessing “place
in therapy” of drugs and other treatments
including cell and gene therapies
• Bridging uncertainties from clinical trials and real-
world performance toward managed
access/performance-based arrangements
• Inclusive of Real-World Evidence requirements
and analysis

10. Patient data: Registries and Real-
World Evidence
• Status of and best practices for patient registries
in Canada and across jurisdictions; core and
disease-specific elements; data-sharing and
privacy
• Real-world evidence as foundational element for
timely, monitored, and cost-effective usage of RD
treatments
Canada Rare Drug Strategy:
Pre-Election Activities
Workstreams cont...
Value-based pricing of Rare Disease
treatments
• Realistic picture of drug prices and costs in
Canada across private and public drug plans and
what this should look like
• Realistic assessment of Canadian drug prices
within OECD countries and where we want to be
• How to strike the balance of timely access, value-
based pricing, and sustainability of healthcare
investment

11. © 2020 3Sixty Public Affairs Inc.
Political situation
• Summer election call nearly a certainty despite
PM’s consistent denials
• Campaign teams, riding nominations, farewell
speeches by exiting MPs, lawn signs ordered;
COVID trends heading in the right direction
• Potential election timing: week of Aug 16/23 ->
election Sept 27 or Oct 4
• Election calculus complicated & campaigns matter:
• Liberals aim to make gains in BC and Ontario and
hold current seats in Quebec
• NDP / Jagmeet Singh’s support rising which
could split the left
• Conservatives / O’Toole have higher “negatives”
than other parties, but have not yet been in
spotlight
11

12. © 2020 3Sixty Public Affairs Inc.
Election issues / positions that could be relevant
12
• National Pharmacare
• Canada Drug Agency
• Rare Diseases Drug
Strategy ($500m/yr)
• Rare Diseases
Treatments
($500M/yr
provinces-2019)
• Reform PMPRB
• Boost
Canada
Health
Transfer
• “New Deal” that
includes
pharmacare
• National
Pharmacare -
basic formulary
(2019)

13. © 2020 3Sixty Public Affairs Inc.
What will the ballot box issue be?
13
• COVID pandemic response?
• Economic recovery?
• Deficits?
• Affordability (housing, gas, food,
pharmacare, child care etc.)?
• Environment?
Source (graph): Nanos – National survey released July 2021. Nanos Research, RDD dual frame hybrid telephone and online random survey, June 30th to July
5th, 2021, n=1051, accurate 3.1 percentage points plus or minus, 19 times out of 20.
Most important policy that will influence vote
QUESTION: What will be the most important policy issue that will influence
your vote if a federal election happens this coming Fall? [RANDOMIZE]
?

14. Challenge for today’s town hall – how to
translate CORD’s key asks into a high-
impact election plan?
Jumpstart Canada’s Rare
Disease Drug Policy (2021)
1. Patient Empowerment
2. Canadian Rare Drug Agency
3. R&D incentives
4. Speed access to treatment
5. Regulatory barriers
6. Regulatory approvals process
7. Pathways for special cases
8. Multiple funding options
9. Managed Access Programs
10. Concurrent HC/HTA reviews
11. Real world evidence
12. Centres of clinical expertise
Canada’s Rare Disease
Strategy (2015)
1. Improving early detection
and prevention
2. Providing timely,
equitable and evidence-
informed care
3. Enhancing community
support
4. Providing sustainable
access to promising
therapies
5. Promoting innovative
research
What CORD can
propose for the
federal election?

15. Other key federal issues: update
• Rare Disease Drug Strategy “What we heard”
report – expected imminently from Health Canada
with more meetings to follow
• PMPRB changes delayed a 3rd time in June 2020,
again in context of COVID pandemic:
—New implementation date now January 1, 2022
—After second delay, PMPRB launched
communications / advocacy strategy; proposed
reduced compliance timelines, then reversed
—After this recent delay, PMPRB proposed
adding new price reductions to reference country
tests and speeding compliance timelines again –
CONSULTATION OPEN UNTIL AUG 15/21

16. • Peter Brenders, BeiGene
• Alexandra Chambers, Novartis
• Cathy Evanochko, Tuberous Sclerosis Canada (TSCST)
• Angela Genge, The Neuro
• Fred Horne, 3Sixty Public Affairs
• Leanne Ward, CHEO
Moderators:
Durhane Wong-Rieger, CORD
Bill Dempster, 3Sixty Public Affairs
CORD’s Mid-summer Super-
panel and Town hall!
Panel